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Alnylam Sees Opportunity in Turning Genes On, And Off

A lot has been written about how Alnylam Pharmaceuticals, and plenty of other companies, see a gold mine in turning off problem genes through drugs based on the biological phenomenon called RNA interference, or RNAi. It turns out that Alnylam (NASDAQ: ALNY), the highflying Cambridge, MA-based biotech company built around RNAi, sees another big opportunity in doing the exact opposite—turning on certain genes that can help combat disease.

The new field is called RNA activation, or RNAa for short. Just like it did when it corralled intellectual property for its bread-and-butter RNAi business, Alnylam took the strategy of looking at the state-of-the-art and grabbing technology licenses that would give it a dominant position in the emerging field, says CEO John Maraganore. Last month, Alnylam said it obtained what it considers the field’s seminal IP from the Salk Institute, the University of Texas Southwestern Medical Center in Dallas, and the University of California, San Francisco.

The fundamental idea here is that sometimes people lack enough of a good protein to stay healthy. For example, researchers would like to know what would happen to people with cancer if a drug could stimulate production of the P53 protein, a well-known tumor suppressor, or P21, a protein that acts as a “stop signal” for cell division. Or, what would happen if researchers could develop a drug to activate a gene that’s otherwise faulty for patients with a single gene defect, like the one that causes cystic fibrosis, a fatal lung disease among children and young adults?

“This is the exact opposite of what we do with RNAi,” says Maraganore. “All diseases are from an overexpression or an underexpression of proteins. When you have an underexpression of certain proteins, that’s where RNAa can be applied.”

The opportunity for RNAa isn’t as broad as with the company’s original gene-silencing technology, because more diseases are caused when the body has too much of a certain physiological activity going on, Maraganore says. RNAa work is still in earliest scientific stages, yet the strategy has impressed Wall Street. … Next Page »